Benitec Biopharma ( (BNTC) ) has provided an update.
Benitec Biopharma Inc. announced promising interim results from its Phase 1b/2a trial for BB-301, showing significant improvements in swallowing function for two subjects with Oculopharyngeal Muscular Dystrophy (OPMD). The low-dose treatment led to clinically meaningful enhancements in swallowing, with one subject achieving a normal swallowing profile. This gene therapy, based on Benitec’s proprietary ddRNAi platform, offers a novel approach to treating OPMD by silencing mutant gene expression while introducing functional protein replacements.
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